OP0042 LONG-TERM EFFICACY AND SAFETY OF CANAKINUMAB IN PATIENTS WITH FAMILIAL MEDITERRANEAN FEVER (FMF) - INTERIM ANALYSIS OF THE RELIANCE REGISTRY
Authors: J. Henes, J. B. Kuemmerle-Deschner, T. Krickau, T. Kallinich, F. Dressler, G. Horneff, F. Meier, I. Foeldvari, F. Weller-Heinemann, B. Kortus-Goetze, M. Hufnagel, J. Rech, P. Oommen, J. Weber-Arden, N. Blank
Journal: Annals of the Rheumatic Diseases (2022)

Background Familial Mediterranean Fever (FMF) is a chronic disease characterized by recurrent attacks of fever as well as serositis and bears the risk of serious complications (e. g. amyloidosis). Treatment of FMF according to EULAR aims to control acute attacks and subclinical inflammation as well as to improve patient´s quality of life 1 . Clinical data indicate that the inhibition of interleukin-1β with canakinumab (CAN) is effective in controlling and preventing flares in FMF patients 2 . Objectives The present study explores the long-term efficacy and safety of canakinumab in routine clinical practice conditions in pediatric (age ≥2 years) and adult FMF patients. Methods RELIANCE is a prospective, non-interventional, multi-center, observational study based in Germany with a 3-year follow-up period. Patients with clinically confirmed FMF diagnosis who routinely receive canakinumab are enrolled in order to evaluate effectiveness and safety of canakinumab. Disease activity and remission by physicians´ assessment, disease activity, fatigue and impact on social life by patients’ assessment, inflammatory markers and AIDAI (Auto-Inflammatory Diseases Activity Index) score were recorded at baseline and assessed at 6-monthly intervals within the 3-year observation period of the study. Results This interim analysis of FMF patients (N=74) enrolled by December 2021 includes baseline as well as 6- to 24-month data. Mean age in this cohort was 25 years (2−61 years) and the proportion of female patients was 51 % (N=38). At baseline, median duration of prior CAN treatment was 1.0 years (0−6 years). At month 24, physician ratings report around 63% of patients in disease remission and patient-reported disease activity (mean PPA) decreased from moderate (3.0) to low (2.6) during the observation period. Other disease activity parameters also decreased (Table 1). A total of 18 serious adverse events were reported, of which 2 (1 case of tonsillectomy and 1 case of tachycardia) were classified as drug - related. Table 1. Baseline characteristics and 4 th interim analysis data of patients with FMF Baseline 12 months 24 months Number of patients, N 74 46 24 Number (%) of patients with days absent from work/school during last 6 months 6 (8) 11 (24) 9 (38) Number (%) of patients in disease remission (physician assessment) 22 (45) 23 (72) 12 (63) Patient’s assessment of current disease activity; 0–10, median (min; max) 2.0 (0; 10) 2.0 (0; 7) 2.0 (0; 10) Patient’s assessment of current fatigue; 0–10, median (min; max) 5.0 (0; 10) 2.0 (0; 10) 4.0 (0; 10) Number (%) of patients without impairment of social life by the disease 27 (50) 28 (80) 8 (67) CRP (mg/dl) | SAA (mg/dl) | ESR (mm/h); median 0.2 | 0.7 | 8.0 0.2 | 0.5 | 4.0 0.2 | 0.7 | 6.0 Number (%) of patients with disease-related symptoms prior to inclusion into the study | at baseline 12 months 24 months Fever 68 (93) | 14 (29) 8 (25) 3 (16) Abdominal pain 67 (92) | 20 (41) 10 (31) 4 (21) Thoracic pain 45 (62) | 5 (10) 3 (9) 1 (5) Headache 34 (47) | 11 (22) 7 (22) 5 (26) Myalgia 23 (32) | 6 (12) 4 (13) 2 (11) Arthralgia/arthritis 39 (54) | 16 (33) 9 (28) 5 (26) Dermal symptoms (urticarial, maculopapulose) 15 (21) | 5 (10) 3 (9) 0 (0) SAE Number of events Incidence rate # per 100 patient years All types of SAE 18 14.03 SADR 2 1.56 Incidence rate = number of events * 36,525 / sum of observation days (=46,848). CRP, c-reactive protein; ESR, erythrocyte sedimentation rate; n. a., not annotated; SAA, serum amyloid A; SADR, serious adverse drug reaction; SAE, serious adverse events. Conclusion Interim data of FMF patients from the RELIANCE study, the longest running real-life canakinumab registry confirm efficacy and safety of long-term canakinumab treatment. References [1]Ozen S, et al. EULAR recommendations for the management of familial Mediterranean fever. Ann Rheum Dis 2016;75:644–651. doi:10.1136/annrheumdis-2015-208690 [2]De Benedetti F, et al. Canakinumab for the treatment of autoinflammatory recurrent fever syndromes. N Engl J Med 2018;378:1908–19. Disclosure of Interests Jörg Henes Consultant of: Novartis, AbbVie, Sobi, Roche, Janssen, Boehringer-Ingelheim, Grant/research support from: Novartis, Roche, J. B. Kuemmerle-Deschner Consultant of: Novartis, AbbVie, Sobi, Grant/research support from: Novartis, AbbVie, Sobi, Tobias Krickau Speakers bureau: Novartis, Consultant of: Novartis, Grant/research support from: Novartis, Tilmann Kallinich Consultant of: Sobi, Novartis, Roche, Grant/research support from: Novartis, Frank Dressler Consultant of: Abbvie, Mylan, Novartis, Pfizer, Grant/research support from: Novartis, Gerd Horneff Speakers bureau: AbbVie, Bayer, Chugai, Merck Sharp &amp; Dohme, Novartis, Pfizer, Roche, Grant/research support from: AbbVie, Chugai, Merck Sharp &amp; Dohme, Novartis, Pfizer, Roche, Florian Meier Speakers bureau: Novartis, Ivan Foeldvari Consultant of: Novartis, Hexal, Medac, Pfizer, Frank Weller-Heinemann: None declared, Birgit Kortus-Goetze Consultant of: Novartis, Markus Hufnagel Grant/research support from: Novartis, Jürgen Rech Speakers bureau: Abbvie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD; Mylan, Novartis, Roche, Sanofi, Sobi, UCB, Consultant of: Abbvie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD, Mylan, Novartis, Roche, Sanofi, Sobi, UCB, Grant/research support from: Novartis, Sobi, Prasad Oommen Grant/research support from: Novartis, Julia Weber-Arden Employee of: Novartis, Norbert Blank Consultant of: Novartis, Sobi, Lilly, Pfizer, Abbvie, BMS, MSD, Actelion, UCB, Boehringer-Ingelheim, Roche, Grant/research support from: Novartis, Sobi.